Encapsulating the Full Impact of Emerging Drugs on Pharma, Patients, and the Global Market

Abstract

Pharmacoeconomic analyses of emerging and existing therapies have been a longstanding mainstay of the content highlighted each month in Clinical Therapeutics. Whether new drug reviews of recently approved therapies or comprehensive pharmacoeconomic assessments on existing treatments for both their value and impact on overall quality of life (quality-adjusted life-years) for patients, these overviews are integral to our understanding of the full impact a drug may have on disease. As emerging therapies continue to push the envelope of technological limits, albeit through gene editing or targeted antibody therapies, access to and cost of a drug may make a lifesaving therapy out of reach for many across the globe. This month, we are excited to launch a new feature in Clinical Therapeutics: the New Drug Capsule. Throughout the year, we will highlight recently approved drugs that have emerged on the market, reviewing their mechanisms of action, targeted disease(s), and population(s), as well as their costs. Our aim is to provide overviews of newly approved therapies to be considered against a broader context of the multiple stakeholder perspectives that may influence the true impact of a drug.For our inaugural New Drug Capsule, we have chosen to highlight etranacogene dezaparvovec (trade name Hemgenix®), a one-time gene therapy recently approved by the US Food and Drug Administration that was developed to treat adults with hemophilia B, a genetic clotting disorder caused by a deficiency of factor IX.1Beninger P. New Drug Capsule: etranacogene dezaparvovec.Clin Ther. 2023; 45 (XXX-XXX)Google Scholar Produced by CSL Behring (King of Prussia, PA, USA), it is notably the most expensive drug in the world, with a price tag of $3.5 million per dose. Over the course of the next months to years, the field will gain important insight into the global access, value, influence of pharmaceutical marketing, and the overall long-term safety of the treatment. We believe the New Drug Capsule summaries will alert our readers to emerging therapies, such as Hemgenix, and will serve as a valuable adjunct to our traditional pharmacoeconomic reviews, including this month's publication by Morimoto et al2Morimoto T Fujito K Yamasaki B et al.Cost-effectiveness analysis of monoclonal antibodies as first-line treatment for RAS wild-type metastatic colorectal cancer: a systematic review.Clin Ther. 2023; 45 (XXX-XXX)Google Scholar that examines the cost-effectiveness of monoclonal antibodies (mABs) as a first-line treatment for RAS wild-type metastatic colorectal cancer. The sticker shock of targeted mABs does not rise to the level of emerging gene therapies. Nevertheless, their costs far exceed more traditional chemotherapeutic agents, questioning their value as a first-line therapy.2Morimoto T Fujito K Yamasaki B et al.Cost-effectiveness analysis of monoclonal antibodies as first-line treatment for RAS wild-type metastatic colorectal cancer: a systematic review.Clin Ther. 2023; 45 (XXX-XXX)Google Scholar Following a systematic review, Morimoto et al concluded that although quality-adjusted life-years and progression-free survival are improved with the use of mABs, traditional chemotherapy alone was cost-effective. The authors further challenge the field to work toward identifying treatment algorithms based on biomarker and/or genetic factors to determine the best therapeutic paths for patients. Undoubtedly, personalized medical approaches will lead to improved outcomes, but here, too, one must be cognizant of patient access to diagnostic platforms required for appropriate risk stratification and the added costs of such assessments. As the New Drug Capsule evolves over time, the diagnostic approaches required for personalization of therapies will be considered in order to encapsulate all the essential contributing factors that influence a drug's success in reaching the most important stakeholder of all—the patient.